Rare Diseases and Orphan Drugs

Produk Detail:
  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Pages : 380 pages
  • ISBN : 9780124199880
  • Rating : /5 from reviews
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Download or Read online Rare Diseases and Orphan Drugs full in PDF, ePub and kindle. this book written by Jules J. Berman and published by Academic Press which was released on 02 June 2014 with total page 380 pages. We cannot guarantee that Rare Diseases and Orphan Drugs book is available in the library, click Get Book button and read full online book in your kindle, tablet, IPAD, PC or mobile whenever and wherever You Like. Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease process manifested in a certain rare disease is strikingly similar to the disease process observed in a common disease. This work ties the lessons learned about rare diseases to our understanding of common ones. Chapters covering the number of common diseases are minimized, while rare diseases are introduced as single diseases or as members of diseases classes. After reading this book, readers will appreciate how further research into the rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases, rare or common. Makes rare diseases relevant to clinicians and researchers by tying lessons learned about the rare diseases to our understanding of the common diseases Stresses basic pathologic mechanisms that account for human disease (e.g., disorders of cell development, replication, maintenance, function and structure), that can be understood without prior training in pathology Discusses advanced concepts in molecular biology and genetics in a simple, functional context appropriate for medical trainees and new researchers Offers insights into how further research into rare diseases may lead to new methods for preventing, diagnosing, and treating all diseases.

Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs
  • Author : Jules J. Berman
  • Publisher : Academic Press
  • Release : 02 June 2014
GET THIS BOOK Rare Diseases and Orphan Drugs

Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often

Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018

Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018
  • Author : ConferenceSeries
  • Publisher : ConferenceSeries
  • Release : 15 June 2021
GET THIS BOOK Proceedings of 4th World Congress on Rare Diseases and Orphan Drugs 2018

June 11-12, 2018 | Dublin, Ireland Key Topics : Neglected Tropical Diseases, Rare Pulmonary Diseases, Rare Diseases in Neurology, Rare Genetic Diseases, Scope of Orphan Drugs, Rare diseases of Endocrine System, Rare diseases of Immune System, Rare Cardiac Diseases, Rare Eye and Ear Diseases, Orphan Drugs Treatment for Rare Diseases, Rare Oral Diseases, Rare Hepatic Diseases, Rare Gastrointestinal Diseases, Rare Bacterial, Viral and Fungal infections, Rare diseases of Genitourinary System, Rare diseases in Nephrology, Rare Skin Diseases, Clinical Research on Orphan Drugs, Rare

Orphan Drugs and Rare Diseases

Orphan Drugs and Rare Diseases
  • Author : David C Pryde,Michael J Palmer
  • Publisher : Royal Society of Chemistry
  • Release : 30 July 2014
GET THIS BOOK Orphan Drugs and Rare Diseases

Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan

Orphan Drugs

Orphan Drugs
  • Author : Elizabeth Hernberg-Ståhl,Miroslav Reljanović
  • Publisher : Elsevier
  • Release : 15 November 2013
GET THIS BOOK Orphan Drugs

This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar

Rare Diseases Epidemiology Update and Overview

Rare Diseases Epidemiology  Update and Overview
  • Author : Manuel Posada de la Paz,Domenica Taruscio,Stephen C. Groft
  • Publisher : Springer
  • Release : 06 December 2017
GET THIS BOOK Rare Diseases Epidemiology Update and Overview

The fields of rare diseases research and orphan products development continue to expand with more products in research and development status. In recent years, the role of the patient advocacy groups has evolved into a research partner with the academic research community and the bio-pharmaceutical industry. Unique approaches to research and development require epidemiological data not previously available to assist in protocol study design and patient recruitment for clinical trials required by regulatory agencies prior to approval for access by

Value Assessment of Orphan Drugs and Treatments for Rare Diseases

Value Assessment of Orphan Drugs and Treatments for Rare Diseases
  • Author : Catherine M. Lockhart
  • Publisher : Unknown
  • Release : 15 June 2021
GET THIS BOOK Value Assessment of Orphan Drugs and Treatments for Rare Diseases

OBJECTIVES: In 1983 the US Orphan Drug Act was passed to facilitate commercialization of drugs to treat rare diseases. The market value for orphan drugs in the US reached $90 billion annually in 2014, with worldwide sales forecast at $176 billion. Payers and policymakers need robust methodology for evaluation of health technology in this growing landscape of expensive treatments for rare diseases. Here I present a systematic review of current practices in value-based evaluation of orphan drugs from a global perspective. I also propose

Rare Diseases and Orphan Products

Rare Diseases and Orphan Products
  • Author : Institute of Medicine,Board on Health Sciences Policy,Committee on Accelerating Rare Diseases Research and Orphan Product Development
  • Publisher : National Academies Press
  • Release : 03 April 2011
GET THIS BOOK Rare Diseases and Orphan Products

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Rare Diseases in the Age of Health 2 0

Rare Diseases in the Age of Health 2 0
  • Author : Rajeev K. Bali,Lodewijk Bos,Michael Christopher Gibbons,Simon Ibell
  • Publisher : Springer Science & Business Media
  • Release : 21 October 2013
GET THIS BOOK Rare Diseases in the Age of Health 2 0

This text focuses on various factors associated with orphan diseases and the influence and role of health information technologies. Orphan diseases have not been adopted by the pharmaceutical industry because they provide little financial incentive to treat or prevent it. It is estimated that 6,000-7,000 orphan diseases exist today; as medical knowledge continues to expand, this number is likely to become much greater. The book highlights the opportunities and challenges in this increasingly important area. The book explores new avenues

The Social Construction of the Orphan Drug Industry

The Social Construction of the Orphan Drug Industry
  • Author : Qing Ying Low (Timothy)
  • Publisher : Unknown
  • Release : 15 June 2021
GET THIS BOOK The Social Construction of the Orphan Drug Industry

"Sociologists have noted that markets are not always formed "naturally", and the creation of markets would require social actors such as the legislator to legitimate it and policies by the state to support its establishment. Using legitimation and choice-within-constraints framework, this would also seem to be the case for the orphan drug industry. Due to the nature of orphan drug being used to treat a small population of patients with rare diseases, legislators in the United States have created new

Physicians Guide to Rare Diseases

Physicians  Guide to Rare Diseases
  • Author : Jess G. Thoene,National Organization for Rare Disorders
  • Publisher : Dowden Publishing Company
  • Release : 15 June 1995
GET THIS BOOK Physicians Guide to Rare Diseases

This classic reference, now with almost 900 disease descriptions, is edited by nationally known pediatrician Jess G. Thoene, M.D. & provides a quick, organized approach to identifying puzzling & unusual symptoms, giving patients the opportunity for early diagnosis & treatment. Special features include: * A full color atlas of visual clues, cross referenced to disease listings. * Thirteen separate specialty sections with an expert overview to introduce each section. Eminent clinicians from the outstanding research centers in rare diseases give you the current status of